Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to ...

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...

Elevidys, Sarepta's gene therapy for DMD, has emerged as the company's primary growth engine. Following its label expansion to include patients aged 6 years and older, the therapy has seen ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

With Pfizer no longer in the hunt, Regenxbio now has a clearer path to becoming Sarepta’s first competitor in the DMD gene therapy market. In November, the company announced that a phase 1/2 ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved gene therapy for DMD, which delivers a micro-dystrophin gene via an ...

Sarepta Therapeutics' Elevidys for DMD shows growing revenue potential, with management targeting >$3bn of total revenues in 2025, up 68% from 2024, led by gene therapy. Despite past valuation ...

Sarepta (SRPT) announced topline results from Part 2 of EMBARK, a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study ...